International MPN News, Science & Opinion

TSR…Summer, 2013


(TSR) The Senyak Report

Simtuzumab – the cocktail clinical trial… The new model patient support meeting… Sanjay Gupta likes weed… Hitch a medical ride with Southwest… Sunshine Law takes effect for docs…  Find a Cure $100 offer


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Simtuzumab — The mixed bag that is Clinical Trial

Clinical Trials are never our first option. And those Phase I/II trials where they test dosage and toxicity on us are truly medieval. Phase III trials where we have a shot at a drug with some proven efficacy is a little better…except who knows whether we’ll be on the placebo arm and get sugar pills for months. But the current Phase II trial of simtuzumab has a few interesting wrinkles.

If you’re already on Jakafi, it might make sense for you and your doctor to check out this clinical trial testing simtuzumab (GS-6624). Simtuzumab an anti-LOXL2 monoclonal antibody is designed to reverse fibrosis. Now a Gilead Sciences drug it’s being combined with Jakafi in one of those “cocktail” trials where a combination of drugs is employed to roll back a disease.

Stephen OhWe talked to Dr. Stephen Oh, in the Division of Hematology at the Washington University (St. Louis) cancer center, one of the trial sites. Dr. Oh, Principal Investigator of the trial, said “What I tell patients is that this is a drug which has the potential to result in improvement in {bone marrow] fibrosis.”

The trial is open to MF patients with stable counts who are already on an established Jakafi regimen. On successful completion, patients would receive the drug without cost as long as there is evidence of clinical benefit.

The trial is recruiting and there are currently three participants at the Washington University site. One likely reason the study isn’t being populated quickly is inconvenience. Patients are required to visit the Washington University facility once every two weeks for an infusion session lasting approximately 30 minutes. Additionally, this is a Phase 2 efficacy and safety trial .

On the upside, there is no placebo arm. Everyone gets the drug. Except for the Jakafi component which patients are required to acquire on their own. It’s sort of a BYOJ party.

There are eight sites in the US currently recruiting, three featuring MPNclinic doctors Mesa, Verstovsek and Gotlib as Principal Investigators. For details, go to www.clinicaltrial.gov, NCT01369498. Accrual contact for the Washington University trial is : Karyn Gordon 314-362-0156 kgordon@dom.wustl.edu

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The new model for patient support meetings..

Here’s one MPN Town Hall meeting not to miss. Saturday, November 23 at MD Anderson in Houston. Check in at 11, meet the panelists and patients around a free box lunch and then head into the session. All FREE, courtesy of PatientPower.info in association with the patient education department at MD Anderson.

And if you can’t make it – that Thursday is Thanksgiving – all is not lost since Andrew Schorr  (shown here with his daughter, Ruthie) Andrew with Ruthieand his crew are planning “lots of video segments” to fill us in.  It wouldn’t be an MD Anderson MPN meeting without Dr Srdan Verstovsek but he won’t be there. He’s off to a meeting in Europe but will participate via Skype and respond to e-mailed questions.

Live at the meeting, presenting updated pre-ASH reports and fielding questions, will be MPN’s most famous hematologist, Dr Ruben Mesa along with practicing clinicians,  MPN nurses and social workers all on hand to field questions and lead break-out sessions.  Andrew will be hosting the event.

After all our public complaints over profit-making, fund-raising MPN patient support events, we’re speechless. This well might be the model of the future. And while there will be no drug company representation at the meeting, Big Pharma will be picking up the tab for the proceedings.

There’s a website coming on-stream where you can get updates and register for the event: http://www.patientpower.info/health-topic/myeloproliferative-disorders

Bonus feature:   Launch of  Dr. Srdan Verstovsek’s new publishing project

And while you’re at the Town Hall meeting pick up one of the first copies of a new newsletter from the  Clinical Research Center for Myeloproliferative Neoplasia   It’s a patient-centered publishing project spearheaded by Dr Verstovsek focused on current research, patient case studies, and expert advice from MD Anderson docs.  Editor is Kate Newberry and she can be reached here kjnewber@mdanderson.org  if you’re interested in submitting stories or other content,

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Sanjay Gupta Likes Weed

That’s the Daily Beast headline to a story covering the pilgrimage of the famed neurosurgeon. Readers were introduced to the subject two years ago in David Ensee’s background story of his own use of marijuana as a PV patient. Dr. Gupta’s long journey and conversion is of special interest to MPN patients seeking symptomatic relief from the disease and treatment.  Once opposed to legalization or use of marijuana, Dr. Gupta has become a strong supporter of medical marijuana. Based on his year’s global research and  direct experience of seeing patients benefit from pot, Dr. Gupta, CNN’s chief medical correspondent, produced this documentary, Weed which aired this month on CNN.

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Sunshine law takes effect… sorta

See that big yellow thing in the sky?  It’s been up there since August 1. It’s  the sun trying to break through the conflict of interest clouds mucking up the medical horizon.  The Physician Payments Sunshine Act  requires drug companies and manufacturers of medical devices to report payment and items of value given to docs and their teaching hospitals.   Healthcare providers are  required to report their sources of funding as one part of the Patient Protection and Affordable Care Act that’s taken effect…despite multiple Congressional efforts to vote down Obama Care.  The information will be publicly available on a searchable website

Implementation is a bit rocky and we won’t actually see results for another year.  Although drug companies and other affected manufacturers began collecting these data August 1, the deadline for affected parties to report to the government isn’t until March 31, 2014.  First disclosure on a public website is scheduled for September 30, 2014. about five weeks before the big mid-term US Election…so maybe we shouldn’t put away our umbrellas just yet.

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Been on a trial the past six months?…$100 for MPNs

Find A Cure Panel is looking for people to participate in a 30 – 45 minute anonymous and confidential telephone call regarding their recent participation in a clinical trial for MPN.  To qualify, you must have participated in a clinical trial for MPN in the last 6 months. For those who participate, FACP will donate $100 to the MPN non-profit of your choice.  To participate, email info@findacurepanel.com and reference QUAL research /MPN Forums.

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Southwest Airlines Medical Transportation Grant Program

The costs of travel to a medical facility can affect our choice of treatment options.  Southwest Airlines has a generous program  to assist patients by providing free flights to many major medical centers. The details are here The page also offers additional sources for flight and travel assistance. There’s a Southwest blog that provides stories and background, here.  Thanks to Sheridan Emery for the tip.

© MPNforum, LLC and MPNforum.com, 2013. MPNforum is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 3.0 Unported License. Unauthorized use and/or duplication of this material without express and written permission is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to MPNforum.com with appropriate and specific direction to the original content.

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